Volume 92; Issue 4

3

A review of the role of enhanced apoptosis in the pathophysiology of cystinosis

Year:
2007
Language:
english
File:
PDF, 499 KB
english, 2007
11

Lysosomal Disease Network’s “WORLD Symposium”

Year:
2007
Language:
english
File:
PDF, 295 KB
english, 2007
12

Annual WORLD Symposium Schedule

Year:
2007
Language:
english
File:
PDF, 120 KB
english, 2007
13

1 Defective neuronal function in LSDs

Year:
2007
Language:
english
File:
PDF, 77 KB
english, 2007
17

5 Mucolipidoses II and III: A genotype–phenotype emerges

Year:
2007
Language:
english
File:
PDF, 44 KB
english, 2007
21

9 Utilizing the brain microvasculature for CNS therapy

Year:
2007
Language:
english
File:
PDF, 45 KB
english, 2007
25

13 Local and systemic gene therapy for metachromatic leukodystrophy

Year:
2007
Language:
english
File:
PDF, 43 KB
english, 2007
29

17 Current and future therapies for hereditary metabolic disorders

Year:
2007
Language:
english
File:
PDF, 40 KB
english, 2007
30

18 Gaucher disease: Complexity in a “simple” disorder

Year:
2007
Language:
english
File:
PDF, 40 KB
english, 2007
37

25 Gene therapy for the CNS manifestations of Batten disease

Year:
2007
Language:
english
File:
PDF, 45 KB
english, 2007
44

33 Screening of newborns for lysosomal storage diseases

Year:
2007
Language:
english
File:
PDF, 46 KB
english, 2007
46

35 Newborn screening for lysosomal storage diseases—A reality?

Year:
2007
Language:
english
File:
PDF, 46 KB
english, 2007
48

38 A proposed multicenter study of CNS structure and function in MPS I

Year:
2007
Language:
english
File:
PDF, 44 KB
english, 2007
51

41 Preparing for experimental therapeutics in JNCL

Year:
2007
Language:
english
File:
PDF, 42 KB
english, 2007
52

42 Pathogenesis of juvenile Batten disease

Year:
2007
Language:
english
File:
PDF, 42 KB
english, 2007
55

46 A perspective on treatment and cures of Gaucher disease

Year:
2007
Language:
english
File:
PDF, 57 KB
english, 2007
61

52 A unique model of GM1 gangliosidosis

Year:
2007
Language:
english
File:
PDF, 42 KB
english, 2007
66

57 Treatment of MPS: Current status, limitations and future challenges

Year:
2007
Language:
english
File:
PDF, 43 KB
english, 2007
68

60 Molecular and biochemical studies of MPS II patients and families

Year:
2007
Language:
english
File:
PDF, 57 KB
english, 2007
73

67 Pompe disease: Lessons from preclinical studies and future challenges

Year:
2007
Language:
english
File:
PDF, 42 KB
english, 2007
74

68 Diagnosing and treating Pompe disease, long term outcome and risk factors

Year:
2007
Language:
english
File:
PDF, 42 KB
english, 2007
77

71 Niemann-Pick disease, type C: A model for pathophysiology and management of LSDs

Year:
2007
Language:
english
File:
PDF, 41 KB
english, 2007
80

75 The hunter outcome survey (HOS): A registry of mucopolysaccharidosis II patients

Year:
2007
Language:
english
File:
PDF, 42 KB
english, 2007
106

Cover 2 / Ed. Board

Year:
2007
Language:
english
File:
PDF, 538 KB
english, 2007
107

Table of Contents

Year:
2007
Language:
english
File:
PDF, 76 KB
english, 2007
108

Announcement

Year:
2007
File:
PDF, 61 KB
2007
109

Supplement title page

Year:
2007
File:
PDF, 57 KB
2007